3 recruiting, 1 completed, 1 terminated. 5 Phase 1, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 5 | Recruiting 3, Completed 1, Terminated 1 |
| Phase 2 | 2 | Recruiting 2 |
Follow Propionic Acidemia: new trials by email Literature Review of Propionic Acidemia trials Research Report on Propionic Acidemia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| AAVrh10-PCCA Gene Therapy for Propionic Acidemia AAVrh10-PCCA low dose, AAVrh10-PCCA middle dose, AAVrh10-PCCA high dose - Propionic Acidemia | Phase 1 | Recruiting | Mayo Clinic | 9 | 2026-07-20 | 2026-08-21 |
| A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study mRNA-3927 - Propionic Acidemia | Phase 1 / Phase 2 | Recruiting | ModernaTX, Inc. | 50 | 2021-11-09 | 2026-03-23 |
| Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia mRNA-3927 - Propionic Acidemia | Phase 1 / Phase 2 | Recruiting | ModernaTX, Inc. | 77 | 2021-04-15 | 2026-01-22 |
| A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia BBP-671, Placebo - Healthy Volunteers, Propionic Acidemia | Phase 1 | Terminated | CoA Therapeutics, Inc., a BridgeBio company | 79 | 2021-03-25 | 2023-12-21 |
| Anaplerotic Therapy in Propionic Acidemia ornithine alpha ketoglutarate, glutamine, disodium citrate - Propionic Acidemia | Phase 1 | Completed | Nicola Longo | 3 | 2008-07-01 | 2015-01-21 |
Acid-Base Imbalance (2) · Acidosis (2) · Amino Acid Metabolism, Inborn Errors (2) · Genetic Diseases, Inborn (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Hyperammonemia (1) · Methylmalonic acidemia (1) · Muscle Hypotonia (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Nutritional and Metabolic Diseases (5) · Amino Acid Metabolism, Inborn Errors (3) · Genetic Diseases, Inborn (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Nervous System Diseases (1) · Neurologic Manifestations (1) · Neuromuscular Manifestations (1) · Pathologic Processes (1) · Pathological Conditions, Signs and Symptoms (1) · Signs and Symptoms (1)
Glutamic Acid (1) · Glutamine (1) · Sodium Citrate (1) · ornithine alpha-ketoglutarate (1)
ModernaTX, Inc. (2) · CoA Therapeutics, Inc., a BridgeBio company (1) · Mayo Clinic (1) · Nicola Longo (1)
United States (5) · Canada (2) · France (2) · Japan (2) · Netherlands (2) · Saudi Arabia (2) · Spain (2) · United Kingdom (2)
David R. Deyle, MD (1) · Nicola Longo, MD, PhD (1)
PMID 28712602, PMID 36577040, PMID 37875310
R21DK077415 (1)